uniQure Q2 2022 Earnings Report
Key Takeaways
uniQure announced encouraging data from the lower-dose cohort in its Phase I/II study of AMT-130 and the advancement of the U.S. and European regulatory reviews for the marketing applications for etranacogene dezaparvovec in hemophilia B. The company held cash and cash equivalents of $500.5 million as of June 30, 2022.
Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington’s disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF).
Announced postponement of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are not affected and no impact is expected on anticipated data readouts in 2023.
Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene dezaparvovec in hemophilia B.
Received notification that GMP certification will be provided to our Lexington manufacturing facility by the European authorities.
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Forward Guidance
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions.